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Trends in clinical success rates and therapeutic focus
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“Human genetics can provide compelling evidence of causation through approaches such as Mendelian randomization (MR).”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
In the citing paper
“Human genetics can provide a tool by which the causal role of an exposure (such as a complex phenotype and/or a drug target) can be characterized 9 .”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
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“Performed well, such studies called Mendelian randomization (MR) analyses, can provide rigorous evidence of causation 9,10 .”
From Integrating genomics with biomarkers and therapeutic targets to invigorate cardiovascular drug development (2021) · cites this paper as [9]
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“Les médicaments qui se rendent aux tests humains officiels dans les essais de phase 1 et 2 ont également un taux d’échec très élevé de 85-93 %.(15) Les chances qu’un médicament soit véritablement efficace augmentent quelque peu dans les essais de phase 3, parce que le travail préliminaire déjà effectué a éliminé de no…”
BackgroundInfluentialJung, A. R., Shin, S. Y., Kim, M. Y., Ha, U., Hong, S., Lee, J. Y., Kim, S. W., Chung, Y., & Park, Y. H. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25052836 · Cited by 7
“Despite the long timeframe and financial investments in this process, the success rate in clinical development has been low [13].”
BackgroundConformation, virtual cocrystal screening, synthesis and determination of dipyridamole
Yi, D., Dong, Y., Qi, M., Hong, M., Zhu, B., & Ren, G. (2024) · Journal of Molecular Structure · doi:10.1016/j.molstruc.2024.139259 · Cited by 6
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“In addition, most drug candidates fail in the later phases of clinical trials and drug approval [3, 4], which is mostly attributed to insufficient drug-like properties, unacceptable toxicity and lack of clinical efficacy [5].”
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“However, it was shown to be already detrimental to the statistical analysis (see Supplementary Figs.3 and 11 and Supplementary Table13).”
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Gellan Gum Promotes the Differentiation of Enterocytes from Human Induced Pluripotent Stem Cells
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“Discussion According to the FDA, the clinical success rate during novel drug development is less than 10% [35].”
BackgroundRecent trends in interorganizational deal networks in pharmaceutical and biotechnology industries
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Generating Customized Compound Libraries for Drug Discovery with Machine IntelligencePreprint
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“The probability of launch from entry to phase I has also remained below 10% [2].”
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“%) and safety concerns (> 20%), with the rest being strategic realignment, and commercial or operational reasons (7–10).”
Translational PBPK/PD modeling in drug discovery: A CRO perspective
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“…fi culty in identifying eligible patients, and variability in disease progression collectively contribute to low enrollment rates, prolonged study timelines, and compromised data integrity in rare disease trials, ultimately resulting in higher failure rates compared to non-rare disease trials 5,6 .”
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“Sun et al. states that 9 out of 10 clinical trials are not successful, 40–50% of which fail due to lack of efficiency and 30% fail due to toxicity or side effects [13,14].”
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“Development of new anti-cancer drugs takes time, and many substances that show promising results in vitro do not result in approved drugs for clinical use, often due to limited effect in vivo (Harrison 2016; Dowden and Munro 2019).”
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“More importantly, only a small fraction of investigational treatments, qualifying for clinical testing, eventually become part of the therapeutic armamentarium (4).”
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“Thus, the failure rate of drug discovery and development is higher than 90% (Takebe et al. 2018; Dowden and Munro 2019; Hinkson et al. 2020; Sun et al. 2022).”
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“Finally, because the probability that a single candidate drug will win approval is low, 9 drugmakers are loath for a factor under their control—dose being one—to contribute to failure.”
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“Today, the convergence of an innovationfriendly environment, a greater focus on translational science, and the emergence of powerful new therapeutic modalities beyond synthetic small molecules has led to dramatic shifts in approach, a greater focus on transformative therapies, and a gradual, but meaningful, uptick in…”
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“The study of the drug approval process suggests that even of those drugs reaching Phase 2 or 3 clinical trials, up to 79% fail to meet the necessary safety and efficacy profile for approval [14,15].”
BackgroundMachine Learning Pipeline for Molecular Property Prediction Using ChemXploreML
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“At the other end of the timeline, the most recent data examines compounds developed by 30 pharmaceutical firms from early 2015 to the end of 2017, yielding a mean failure rate of 93% (Dowden and Munro, 2019).”
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“Approximately 30 % of clinical failures in drug development have been attributed to unmanageable toxicity [1 – 3].”
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“However, approximately 79% of phase II trials fail to progress due to safety/efficacy issues (Dowden and Munro, 2019), based on the inherent difficulties in extrapolating drug data from animal to human (Basketter et al., 2012).”
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“It is recorded that drug discovery is a cost and time-intensive process with a failure rate of over 90%, taking more than 2 billion US dollars and about 10-15 years on average for an innovative drug to be developed and finally put on the market [2].”
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“According to Helen Dowden, et al. [35] and Richard K. Harrison [36], the failure rate of research and development of new drugs in phase I/II was as high as 90%, of which 40–45% of cases were related to problems of toxicity and drug-like properties.”
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“Successful drug development for central nervous system (CNS) disorders remains a challenge, with clinical failures outpacing successes 1 .”
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Alanazi, A. A. A., Fahad, . I. A. A., Almorshed, A. S. A., Alrbian, A. A. M., Alnughaymishi, A. A. S., Al-Mutairi, N. H. B., Alajmi, A. A., & Otaibi, S. G. A. (2022) · International Journal of Health Sciences · doi:10.53730/ijhs.v6ns10.15290 · Cited by 5
“Furthermore, the probability of progressing a drug from Phase I clinical trials to market is discouragingly low, with a success rate below 10% [2].”
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“The development of anti-cancer drugs or therapies is a long, costly, and high-risk process and nine out of ten candidate drugs or therapies fail in clinical trials (Dowden and Munro, 2019).”
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Metadata
- Authors
- Dowden, H., & Munro, J.
- Corresponding author
- Munro, Jamie
- Venue
- Nature Reviews Drug Discovery
- Published
- 2019-05-08
- Volume
- 18
- Issue
- 7
- Pages
- 495-496
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- OpenAlex
- W2944466104
- Cited by
- 649
- References
- 0
- Citations by year
- 2026: 101 · 2025: 129 · 2024: 140 · 2023: 114 · 2022: 57 · 2021: 57 · 2020: 43 · 2019: 7
- FWCI
- 39.38
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cancer Research › Science, Research, and Medicine
- Health Sciences › Medicine › Pharmacology (medical) › Historical Medical Research and Treatments
- Health Sciences › Medicine › Internal Medicine › General Medicine and Therapeutics
- Keywords
- clinical success rate, therapeutic focus, drug development, clinical trial results, therapeutic areas
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
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